Efficacy of Continuous Interleukin 1 Blockade in Mevalonate Kinase Deficiency: A Multicenter Retrospective Study in 13 Adult Patients and Literature Review.

Authors :: Deshayes S
Georgin-Lavialle S
Hot A
Durel CA
Hachulla E
Rouanes N
Audia S
Le Gallou T
Quartier P
Urbanski G
Messer L
Klein S
de Boysson H
Bienvenu B
Grateau G
Aouba A
Source :: The Journal of rheumatology [J Rheumatol] 2018 Mar; Vol. 45 (3), pp. 425-429. Date of Electronic Publication: 2018 Jan 15.
Publication Year :: 2018
Abstract: Objective: To report efficacy and tolerance of interleukin 1 blockade in adult patients with mevalonate kinase deficiency (MKD).<br />Methods: We retrospectively collected data on 13 patients with MKD who had received anakinra (n = 10) and canakinumab (n = 7).<br />Results: Anakinra resulted in complete or partial remission in 3/10 and 5/10 patients, respectively, and no efficacy in 2/10, but a switch to canakinumab led to partial remission. Canakinumab resulted in complete or partial remission in 3/7 and 4/7 patients, respectively.<br />Conclusion: These data support frequent partial responses, showing a better response with canakinumab. The genotype and therapeutic outcomes correlation should help in the personalization of treatment.

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